to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of escalating single and multiple doses of kp104

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Summary: the purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of kp104 in healthy volunteers. the study will be conducted in 2 parts: part 1, the single ascending dose (sad) is the first in human (fih) study of kp104 and part 2, multiple ascending dose (mad).


Eligibility criteria:
inclusion criteria: * weight of \> 40 kilograms (kg) and \< 120 kg at screening. * in good general health, determined by no clinically significant findings in the opinion of the investigator from medical history, physical examination, 12-lead ecg, clinical laboratory findings, and vital signs at screening and check-in. * hemoglobin, hematocrit, white blood cell count, absolute neutrophil count, and platelet count results within the normal range at the screening visit; participants with gilbert's disease with associated abnormalities of liver function tests are eligible for enrollment. tests may be repeated at the discretion of the investigator to confirm abnormalities. * creatinine clearance based on the cockcroft-gault equation of \>= 80 milliliters per minute (ml/min). * females of childbearing potential and males must practice effective contraception from screening until 28 days after the end of study (eos) visit. * females of childbearing potential must have a negative pregnancy test at screening and within 24 hours prior to dosing of study drug; for post-menopausal subjects, a blood sample will also be tested for follicle stimulating hormone to confirm post-menopausal status. exclusion criteria: * any clinically significant underlying illness in the opinion of the investigator. * any history or sign of significant chronic active or recurrent infection, or screening laboratory evidence consistent with a significant chronic active or recurrent infection requiring treatment with antibacterials, antivirals, or antifungals. * treatment of any infection with iv (within 30 days of screening) or oral (within 14 days of screening) antibacterials, antivirals, or antifungals. * history of clinically significant hematologic or bone marrow disease or blood dyscrasias. * history of meningococcal infection. * history of tuberculosis. * history of asplenia (functional or anatomical). * prior exposure to kp104. * known allergy to penicillin antibiotics or history of allergy or contraindication to required prophylactic antibiotic therapy to be used during the study. * known or suspected complement deficiency during screening. * positive serology for hepatitis b virus (hbv), hepatitis c virus (hcv) or human immunodeficiency virus (hiv) at screening. * history of drug or alcohol abuse within 1 year of screening in the opinion of the investigator, or a positive test for drugs of abuse or alcohol at screening or check-in. note: other protocol defined inclusion/exclusion criteria may apply.

Keywords: kp104 paroxysmal nocturnal hemoglobinuria single ascending dose multiple ascending dose first in human complement inhibitor healthy participants